The U.S. Food and Drug Administration (FDA) granted ONCOFID‑P “orphan drug status” for treating malignant mesothelioma.
ONCOFID‑P is a combination of paclitaxel and hyaluronic acid. Paclitaxel is a chemotherapy drug, often known by brand names Taxol and Abraxane. Hyaluronic acid is a treatment for knee pain, burns and other skin issues. There’s hope the combination can slow, or even stop, the progression of mesothelioma tumors.
Receiving orphan drug status doesn’t mean the FDA will approve ONCOFID‑P in the future. It also doesn’t indicate the therapy is safe or effective for mesothelioma. This designation simply helps the manufacturer and shines more attention toward the therapy as it moves through the clinical trial process.
Why Did the FDA Make ONCOFID‑P an Orphan Drug?
The FDA made ONCOFID‑P an orphan drug for pleural mesothelioma due to preclinical testing. The therapy can bind specifically to cells expressing CD44, a cell‑surface molecule active in cell proliferation. CD44 is highly expressed on the surface of malignant pleural mesothelioma cells.
By binding to these cells, thanks to the hyaluronic acid, a greater amount of paclitalex penetrates the disease rather than affecting healthy cells. The preclinical testing indicates “high anti‑tumor activity,” according to the manufacturer’s press release.
The manufacturer, Fidia Farmaceutici, announced the FDA orphan drug label last week. Fidia is based in Italy.
“The FDA designation represents an opportunity for physicians and patients who, until now, did not benefit from any innovative therapy,” Carlo Pizzocaro, president and chief executive officer of Fidia Farmaceutici, said in the company’s press release. “This result demonstrates that the scientific research must not stop and that every step forward will add hope for patients, especially those who are victims of such serious rare diseases.”
What Is Paclitaxel?
Paclitaxel is the chemotherapy drug used in HIPEC, a type of heated chemoperfusion treatment for peritoneal mesothelioma. It’s also used experimentally for malignant pleural mesothelioma.
The FDA believes paclitaxel isn’t as effective on its own versus with hyaluronic acid. That explains the momentum for ONCOFID‑P.
“In experimental data looking at pleural mesothelioma, CD44‑expressing cells are up to 70 times more susceptible to ONCOFID‑P compared to paclitaxel alone,” reads a report on the Targeted Oncology website.
ONCOFID‑P is also more effective than paclitaxel at reducing tumor size.
ONCOFID‑P is currently in clinical testing for bladder cancer. However, it hasn’t reached the human testing phase for malignant mesothelioma.
What Is FDA Orphan Drug Status?
Orphan drug status is a label for rare diseases, such as mesothelioma. The intent is to inspire development of new therapies for undertreated cancers.
After receiving the designation, the drug sponsor and manufacturer receive incentives, such as tax credits for clinical testing. These incentives help manufacturers and sponsors test therapies to determine if they could improve survival rates.
Malignant mesothelioma doesn’t have many approved therapies. The FDA approved a chemotherapy combination, pemetrexed and cisplatin, nearly two decades ago. The FDA approved a tumor treating fields device for pleural mesothelioma in 2019. In 2020, the FDA approved an immunotherapy combination, Opdivo and Yervoy, for pleural mesothelioma.
The scarcity of safe and effective options is why the FDA grants orphan drug status for mesothelioma therapies.
Sources & Author
- Novel Mesothelioma Treatment Oncofid-P Granted FDA Orphan Drug Status. Targeted Oncology. Retrieved from: https://www.targetedonc.com/view/novel-mesothelioma-treatment-oncofid-p-granted-fda-orphan-drug-status. Accessed: 07/27/2021.
- Fidia Announced the FDA Orphan Drug Designation for ONCOFID for the Treatment of Malignant Mesothelioma, the Cancer Caused by Exposure to Asbestos. Fidia. Retrieved from: https://www.fidiapharma.com/en/news-fidia-announces-the-fda-orphan-drug-designation-for-oncofid-for-the-treatment-of-malignant-mesothelioma-the-cancer-caused-by-exposure-to-asbestos-3-288,3,288. Accessed: 07/27/2021.
- Synergistic Enhancement of Cellular Uptake With CD44-Expressing Malignant Pleural Mesothelioma by Combining Cationic Liposome and Hyaluronic Acid-Lipid Conjugate. Journal of Pharmaceutical Sciences. Retrieved from: https://pubmed.ncbi.nlm.nih.gov/31229434/. Accessed: 07/27/2021.
- Chemotherapy for Malignant Mesothelioma. American Cancer Society. Retrieved from: https://www.cancer.org/cancer/malignant-mesothelioma/treating/chemotherapy.html. Accessed: 07/27/2021.
- Designating an Orphan Drug: Drugs and Biological Products. U.S. Food and Drug Administration. Retrieved from: https://www.fda.gov/industry/developing-products-rare-diseases-conditions/designating-orphan-product-drugs-and-biological-products. Accessed: 07/27/2021.
Sources & Author